A phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with beta-thalassemia.
نویسندگان
چکیده
Deferasirox (ICL670) is a once-daily oral iron chelator developed for the treatment of chronic iron overload from blood transfusions. A comparative phase 3 trial was conducted to demonstrate the efficacy of deferasirox in regularly transfused patients with beta-thalassemia aged 2 years or older. Patients were randomized and received treatment with deferasirox (n = 296) or deferoxamine (n = 290), with dosing of each according to baseline liver iron concentration (LIC). The primary endpoint was maintenance or reduction of LIC; secondary endpoints included safety and tolerability, change in serum ferritin level, and net body iron balance. In both arms, patients with LIC values of 7 mg Fe/g dry weight (dw) or higher had significant and similar dose-dependent reductions in LIC and serum ferritin, and effects on net body iron balance. However, the primary endpoint was not met in the overall population, possibly due to the fact that proportionally lower doses of deferasirox relative to deferoxamine were administered to patients with LIC values less than 7 mg Fe/g dw. The most common adverse events included rash, gastrointestinal disturbances, and mild nonprogressive increases in serum creatinine. No agranulocytosis, arthropathy, or growth failure was associated with deferasirox administration. Deferasirox is a promising once-daily oral therapy for the treatment of transfusional iron overload.
منابع مشابه
Randomized phase II trial of deferasirox (Exjade, ICL670), a once-daily, orally-administered iron chelator, in comparison to deferoxamine in thalassemia patients with transfusional iron overload.
BACKGROUND AND OBJECTIVES Iron accumulation is an inevitable consequence of chronic blood transfusions and results in serious complications in the absence of chelation treatment to remove excess iron. Deferoxamine (Desferal, DFO) reduces morbidity and mortality although the administration schedule of slow, parenteral infusions several days each week limits compliance and negatively affects long...
متن کاملIron chelator drugs in thalassemia,a review article
Abstract Thalassemia syndromes are the most common inherited hemoglobinopathies in the world characterized by various degrees of defective production of the alpha or beta globin chains. Impairment in alpha chain or beta chain synthesis leads to alpha thalassemia and beta thalassemia syndromes respectively. Iran is one of the countries located on the thalassemia belt therefore this disease is ...
متن کاملEfficacy of Deferasirox (Exjade®) versus Osveral® in Treatment of Iron Overload in Patients with Beta- thalassemia Major in Iran; A Non-randomized Controlled Trial
Background: Iron chelators are an important part of management of patients with thalassemia. It is prudent to compare efficacy of different iron chelators in treatment of iron overload in these patients who receive regular blood transfusion. We aimed to compare the efficacy and safety of available oral iron chelator; Deferasirox (Exjade®) with Deferasirox (Osveral®) in reducing iron ove...
متن کاملExjade® (deferasirox, ICL670) in the treatment of chronic iron overload associated with blood transfusion
Although blood transfusions are important for patients with anemia, chronic transfusions inevitably lead to iron overload as humans cannot actively remove excess iron. The cumulative effects of iron overload lead to significant morbidity and mortality, if untreated. Although the current reference standard iron chelator deferoxamine has been used clinically for over four decades, its effectivene...
متن کاملA phase 3 study of deferasirox (ICL670), a once-daily oral iron chelator, in patients with -thalassemia
Maria Domenica Cappellini, Alan Cohen, Antonio Piga, Mohamed Bejaoui, Silverio Perrotta, Leyla Agaoglu, Yesim Aydinok, Antonis Kattamis, Yurdanur Kilinc, John Porter, Marcello Capra, Renzo Galanello, Slaheddine Fattoum, Guillermo Drelichman, Carmelo Magnano, Monica Verissimo, Miranda Athanassiou-Metaxa, Patricia Giardina, Alexandra Kourakli-Symeonidis, Gritta Janka-Schaub, Thomas Coates, Christ...
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عنوان ژورنال:
- Blood
دوره 107 9 شماره
صفحات -
تاریخ انتشار 2006